JERUSALEM, Oct. 17 (Xinhua) -- Israeli drug developer Protalix Biotherapeutics announced on Thursday a success in trials of a drug for the treatment of Fabry disease.
Fabry is a rare hereditary genetic disease where the enzyme activity called alpha-galactosidase is decreased.
As a result, the breakdown of a fatty-sugary substance is damaged, causing its accumulation in blood, vessel, heart, kidney and nerve cells.
The first symptoms appear in childhood and include severe pain in the extremities, intolerance of heat and cold, and lack of sweating.
As age increases, reddish-purple skin lesions are added, especially in the groin and navel area, and great fatigue is felt.
In adulthood there is pain relief, but then signs of irreversible damage to the kidneys, heart and brain begin to accrue.
The new drug, called pegunigalsidase alfa, has been tested on 22 Fabry patients, previously treated with the Replagal, one of the three approved drugs for the disease.
According to the company, after a 12-month treatment with the experimental drug, improvement in the renal function of 16 of the 22 subjects was found.
In addition, the tested drug was found to be well tolerated in the study, with all adverse events being transient in nature without sequelae.
